Clinical Trials

Clinical trials are the primary way researchers determine if a new treatment is safe and effective in people. Additionally, they determine whether the treatment is more effective and/or has less harmful side effects than the current “standard of care” treatments. Currently, the only standard treatment for DIPG/DMG is radiation.

In recent years, clinical trials have provided researchers valuable information about DIPG/DMG. As researchers discover more about DIPG/DMG, they can adjust clinical trials to test new ideas. In the last decade, this information has helped in understanding the biology of DIPG/DMG tumors. Researchers are now optimistic that new and future clinical trials will lead to new standard of care treatments, which will enable much longer survival times and eventually a cure.

Clinical trial availability varies, and not all trials are available in all geographical locations. Inclusion and exclusion criteria must be met before a patient can enroll into a clinical trial. The trial will have an FDA-approved protocol that allows doctors to enroll patients to try certain treatments. Your oncology team may provide you trial options based on geographical availability.

Many of the current trials for DIPG/DMG require a biological understanding of the tumor, which can only be discovered through a biopsy. This biological understanding includes identification of mutations present in the tumor. Studies are now being designed to provide individualized therapy based on specific mutations found in DIPG/DMG.

Clinical trials are conducted in phases

Phase I

A phase I trial is designed to determine whether the drug is safe and to find the maximum tolerated dose for the medication. It is the first trial in humans under the approved plan. The trial drug starts at a very low dose and is increased once the current dose is determined safe. This process usually continues until the maximum tolerated dose is achieved. Phase I trials are usually designed for only a small number of patients. Most DIPG/DMG trials are in phase 1.
Phase II
Phase III

A phase III trial is usually designed to compare the use of a new drug, sometimes paired with another medication, against another treatment or drug. Phase III trials have a larger number of patients enrolled and are conducted at more investigative sites. It is currently unusual to have a phase III trial for DIPG/DMG; however, researchers are getting closer every day.

All clinical research trials will include an Institutional Review Board (IRB) informed consent (IC). The IC will need to be reviewed and signed by a parent or guardian of the patient. The IC will explain the operation of the clinical trial, advise on what you can expect along the way, and provide information about the study medication.

Typically the cost of the medication or agent is covered by the trial when accepted into a clinical trial. However, some medical expenses may be the responsibility of the patient or family. You should have a clear understanding of your financial responsibility prior to entering the trial. Sometimes clinical trials in the United States may not be available to international patients due to individual institutional requirements such as U.S. medical insurance.

Please refer to https://www.clinicaltrials.gov and search for DIPG and/or DMG to view current clinical trials.

It can be difficult to understand exactly which trials are accepting new patients and whether your child qualifies.

Information about clinical trials can also change somewhat frequently. You are not alone as you try to determine the next steps for your family. Our Nurse Navigators are here to help.

Clinical trials are conducted in phases

Phase I

Phase II

Phase III

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